Satellos Bioscience Inc. has announced a change to the primary efficacy endpoint of its BASECAMP Phase 2 pediatric clinical trial evaluating Forazapadin in Duchenne muscular dystrophy (DMD), following feedback from the U.S. Food and Drug Administration (FDA). The company has selected MRI-measured muscle fat fraction as the new primary efficacy endpoint, replacing dynamometry.
The decision follows encouraging findings from the company’s Phase 2 TRAILHEAD study in adults with DMD, which demonstrated a reduction in muscle fat fraction measured by magnetic resonance imaging (MRI) of the biceps brachii muscle.
Over five months of treatment with Forazapadin, participants in TRAILHEAD showed a mean reduction of 3.7 percentage points in MRI-measured biceps brachii muscle fat fraction, decreasing from 49.7 percent at baseline to 46.0 percent on Day 140. This finding indicates an improvement in muscle composition.
Satellos also reported increased total effort, stable muscle strength, and lower creatine kinase (CK) levels in the TRAILHEAD study. The safety and tolerability profile remained consistent with previously reported data. These findings provide additional clinical observations alongside the reduction in MRI-measured muscle fat fraction.
BASECAMP Phase 2 Trial: New Primary Endpoint
The BASECAMP study is a global, randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating Forazapadin in 53 ambulatory boys with DMD aged 7, 8, or 9 years.
The study includes an initial 12-week placebo-controlled treatment period, followed by a 36-week active-treatment period during which all participants receive Forazapadin.
Following FDA feedback, the primary efficacy endpoint will now measure changes in vastus lateralis muscle fat fraction using MRI. Secondary and exploratory endpoints will assess muscle quality, functional outcomes, and biomarkers of muscle regeneration, including Satellos’ proprietary Regenerative Index.
Fat fraction is an objective MRI measurement of the proportion of fat within muscle tissue. In Duchenne, healthy muscle is progressively replaced by fat, making changes in muscle fat fraction an important measure of disease progression and muscle composition.
Satellos expects to report topline results from the 12-week placebo-controlled BASECAMP period in Q1 2027.
Forazapadin is an investigational oral small-molecule AAK1 inhibitor designed to enhance the body’s natural ability to repair and regenerate skeletal muscle. It is being developed as a potential dystrophin-independent treatment for Duchenne muscular dystrophy.
Discover More: Duchenne Clinical Trial Locations Map
